CGTLive®’s Weekly Rewind – July 19, 2024
Review top news and interview highlights from the week ending July 19, 2024.
CGTLive®’s Weekly Rewind
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.
1. CBER To Conduct Interactive CGT Site Tours to Improve Communication, Share Best Practices
The FDA announced its START Pilot Program in October 2023 with a similar goal.
2. David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.
3. Gene Therapy Improves Some Disease Biomarkers of Friedreich Ataxia Cardiomyopathy
Despite the improvements, Lexeo was unable to discern a benefit on cardiovascular fitness and Peak VO2 from LX2006 with current measurements.
4. Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.
5. Dopaminergic Progenitor Cell Therapy Shows Some Symptom Improvement in Parkinson Disease
Data from the high-dose cohort of a phase 1/2 clinical trial in South Korea will be shared in Fall 2024.
